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Background: Advanced methods for causal inference, such as targeted maximum likelihood estimation (TMLE), require certain conditions for statistical inference. However, in situations where there is not differentiability due to data sparsity…

Randomized Controlled Trials (RCTs) are often considered the gold standard for estimating causal effect, but they may lack external validity when the population eligible to the RCT is substantially different from the target population.…

Methodology · Statistics 2023-01-11 Bénédicte Colnet , Julie Josse , Erwan Scornet , Gaël Varoquaux

We address estimation of intervention effects in experimental designs in which (a) interventions are assigned at the cluster level; (b) clusters are selected to form pairs, matched on observed characteristics; and (c) intervention is…

Methodology · Statistics 2014-11-24 Zhenke Wu , Constantine E. Frangakis , Thomas A. Louis , Daniel O. Scharfstein

In certain privacy-sensitive scenarios within fields such as clinical trial simulations, federated learning, and distributed learning, researchers often face the challenge of estimating correlations between variables without access to…

Methodology · Statistics 2025-08-05 Longwen Shang , Min Tsao , Xuekui Zhang

We introduce R package iglm, which implements a comprehensive framework for studying relationships among predictors and outcomes under interference. The implemented regression framework facilitates the study of spillover and other phenomena…

Computation · Statistics 2026-05-05 Cornelius Fritz , Michael Schweinberger

Estimation of local average treatment effects in randomized trials typically requires an assumption known as the exclusion restriction in cases where we are unwilling to rule out unmeasured confounding. Under this assumption, any benefit…

Methodology · Statistics 2020-08-17 Andrew J. Spieker , Robert A. Greevy , Lyndsay A. Nelson , Lindsay S. Mayberry

Evaluations often inform future program implementation decisions. However, the implementation context may differ, sometimes substantially, from the evaluation study context. This difference leads to uncertainty regarding the relevance of…

Methodology · Statistics 2023-12-14 Irina Degtiar , Mariel Finucane

Latent Markov (LM) models represent an important class of models for the analysis of longitudinal data (Bartolucci et. al., 2013), especially when response variables are categorical. These models have a great potential of application for…

Computation · Statistics 2015-01-20 Francesco Bartolucci , Alessio Farcomeni , Silvia Pandolfi , Fulvia Pennoni

Non-negative two-part outcomes are defined as outcomes with a density function that have a zero point mass but are otherwise positive. Examples, such as healthcare expenditure and hospital length of stay, are common in healthcare…

A targeted learning (TL) framework is developed to estimate the difference in the restricted mean survival time (RMST) for a clinical trial with time-to-event outcomes. The approach starts by defining the target estimand as the RMST…

Methodology · Statistics 2026-01-21 Man Jin , Yixin Fang

Analyzing data from multiple sources offers valuable opportunities to improve the estimation efficiency of causal estimands. However, this analysis also poses many challenges due to population heterogeneity and data privacy constraints.…

Methodology · Statistics 2025-10-23 Rong Zhao , Jason Falvey , Xu Shi , Vernon M. Chinchilli , Chixiang Chen

Modern deep neural networks are powerful predictive tools yet often lack valid inference for causal parameters, such as treatment effects or entire survival curves. While frameworks like Double Machine Learning (DML) and Targeted Maximum…

Machine Learning · Computer Science 2025-07-17 Yi Li , David Mccoy , Nolan Gunter , Kaitlyn Lee , Alejandro Schuler , Mark van der Laan

For many health conditions, there are highly efficacious treatment and prevention products. Maximizing their impact requires strategies that improve the reach of health screening in order to establish who could benefit. For example, HIV…

Heterogeneous treatment effect (HTE) estimation is critical in medical research. It provides insights into how treatment effects vary among individuals, which can provide statistical evidence for precision medicine. While most existing…

Machine Learning · Statistics 2025-04-25 Ke Wan , Kensuke Tanioka , Toshio Shimokawa

Frailty models are often the model of choice for heterogeneous survival data. A frailty model contains both random effects and fixed effects, with the random effects accommodating for the correlation in the data. Different estimation…

Methodology · Statistics 2019-09-17 Oodally Ajmal , Luc Duchateau , Estelle Kuhn

Estimating treatment effects (TE) from observational data is a critical yet complex task in many fields, from healthcare and economics to public policy. While recent advances in machine learning and causal inference have produced powerful…

Machine Learning · Computer Science 2025-08-15 Jeroen Berrevoets , Julianna Piskorz , Robert Davis , Harry Amad , Jim Weatherall , Mihaela van der Schaar

Developing new drugs for target diseases is a time-consuming and expensive task, drug repurposing has become a popular topic in the drug development field. As much health claim data become available, many studies have been conducted on the…

Machine Learning · Computer Science 2023-02-22 Yaobin Ling , Pulakesh Upadhyaya , Luyao Chen , Xiaoqian Jiang , Yejin Kim

Personalized decision-making, aiming to derive optimal treatment regimes based on individual characteristics, has recently attracted increasing attention in many fields, such as medicine, social services, and economics. Current literature…

Methodology · Statistics 2023-02-28 Jianing Chu , Wenbin Lu , Shu Yang

In a typical two-phase design, a random sample is drawn from the target population in phase 1, during which only a subset of variables is collected. In phase 2, a subsample of the phase-1 cohort is selected, and additional variables are…

Methodology · Statistics 2026-03-02 Sky Qiu , Susan Gruber , Pamela A. Shaw , Brian D. Williamson , Mark J. van der Laan

Signed graphs encode similarity and dissimilarity relationships among different entities with positive and negative edges. In this paper, we study the problem of community recovery over signed graphs generated by the signed stochastic block…

Social and Information Networks · Computer Science 2022-02-25 Xiaolu Wang , Peng Wang , Anthony Man-Cho So
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